Changes in Bone Mineral Density Following Conventional Oral Phosphonate Treatment of Hypophosphatemic Osteomalacia: A Non-Randomized Controlled Study.

Changes in Bone Mineral Density Following Conventional Oral Phosphonate Treatment of Hypophosphatemic Osteomalacia: A Non-Randomized Controlled Study.
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传统口服磷酸盐治疗低磷血症性骨软化症后骨矿物质密度的变化:一项非随机对照研究

DOI:
10.2147/ijgm.s332534
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发表时间:
2021
影响因子:
2.3
通讯作者:
Zhou HD
Zhou HD
中科院分区:
医学4区
文献类型:
--
作者:
Guo Y;Zhou YH;Wu XP;Tang CY;Wang M;Mo ZH;Shepherd JA;Ng BK;Fan B;Zhou HD

文献摘要

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目的:有有限的临床研究旨在解决口服磷酸盐和骨化三醇在成人低磷酸盐血症性骨软化症(HO)的常规治疗的有效性问题。此外,目前尚无良好的非危险性指标来评价骨软化症治疗前后骨丢失的严重程度。因此,本研究的目的是评估自混合磷酸盐补充剂和骨化三醇对HO患者的常规治疗的疗效,以及骨矿物质密度(BMD)是否有助于监测疗效。患者和方法共纳入21例HO患者和105例健康对照者。所有患者均进行了血清生物标志物和腰椎(L1-L4)、股骨颈和左髋关节的BMD检测。治疗3年后,21例HO患者中有11例被召回进行BMD测量。根据用药情况,将服用钙剂和骨化三醇的HO患者分为3个磷酸盐治疗组:A组(n = 3)持续补充磷酸盐,B组(n = 5)间断补充磷酸盐,C组(n = 3)不补充磷酸盐。结果21例HO患者中,遗传性低磷性佝偻病5例,以肾小管性酸中毒和维生素D缺乏为特征的Fanconi综合征4例,遗传性维生素D异常12例。患者组的平均初始血清磷水平比对照组低约50%。HO组腰椎和全髋的BMD显著低于对照组。与基线相比,磷酸盐补充剂的连续治疗可使腰椎和全髋关节的BMD增加33.4-52.3%,股骨颈增加43.2-79.3%,并且一旦停止治疗,该效果似乎仍在继续。结论常规治疗可改善HO患者骨缺损,尤其是股骨颈骨缺损。骨密度检测是评价骨缺损程度和治疗效果的良好工具。试验注册中国临床试验注册中心,ChiCTR-OOC-16010095。2016年12月7日注册。已登记的逆行。
Purpose There are limited clinical studies aimed at solving the problem of the efficiency of conventional treatment with oral phosphate and calcitriol in adults with hypophosphatemic osteomalacia (HO). In addition, there still had no good non-hazardous markers to evaluate the severity of bone loss of osteomalacia before and after treatment. Therefore, the purpose of this study was to assess the efficacy of conventional treatment with a self-blended phosphate supplementation and calcitriol on patients with HO and whether bone mineral density (BMD) can be helpful for monitoring the efficacy. Patients and Methods A total of 21 HO patients and 105 healthy controls were enrolled. All patients were tested for serum biomarkers and BMD of the lumbar spine (L1–L4), femoral neck, and total left hip. After three years of treatment, 11 of 21 HO patients were recalled for BMD measurement. According to the administration of drugs, HO patients with calcium and calcitriol were divided into three phosphate treatment groups: patients in group A (n = 3) received continuous phosphate supplementation, patients in group B (n = 5) received intermittent phosphate supplementation and patients in group C (n = 3) received no phosphate supplementation. Results The diagnoses of 21 HO patients were 5 cases of hereditary hypophosphatemic rickets, 4 cases of Fanconi syndrome with the features of renal tubular acidosis and vitamin D deficiency, and 12 cases of hereditary vitamin D abnormality. The average initial serum phosphorus level of the patient group was approximately 50% lower than that of the control group. Lower BMD was significantly observed in the HO group than the control group at the lumbar spine and total hip. Continuous treatment with the phosphate supplement could increase BMD in the lumbar spine and total hip by 33.4–52.3% and in the femoral neck increased by 43.2–79.3% compared with baseline, and the effect appears to be continued once treatment is discontinued. Conclusion These findings suggest that conventional therapy can improve bone mineral defects in patients with HO, especially in the femoral neck. Detection of BMD in HO patients is a good tool to assess the extent of bone defects and the therapeutic effect. Trial Registration Chinese Clinical Trial Registry, ChiCTR-OOC-16010095. Registered 7 December 2016. Retrospectively registered.